/Gene Therapy
Abstract

A method of gene therapy in a subject in need thereof, the method comprising administering to the subject a vector that comprises a transgene for expression in the subject and a mirtron for knocking down expression of a gene in the subject, wherein the mirtron is in the 5′UTR of the transgene. Also a vector comprising a transgene for expression from the vector and a mirtron for knocking down expression of a gene, wherein the mirtron is in the 5′UTR of the transgene and wherein the 5′UTR further comprises an exonic splice enhancer motif and/or wherein the mirtron is flanked in the 5′UTR by a fragment of the coding region of a reporter gene, and methods for preparing a vector.

Full Text

What is claimed is:

A method of gene therapy in a subject in need thereof, the method comprising administering to the subject a vector that comprises a transgene for expression in the subject and a mirtron for knocking down expression of a gene in the subject, wherein the mirtron is in the 5′UTR of the transgene. Also a vector comprising a transgene for expression from the vector and a mirtron for knocking down expression of a gene, wherein the mirtron is in the 5′UTR of the transgene and wherein the 5′UTR further comprises an exonic splice enhancer motif and/or wherein the mirtron is flanked in the 5′UTR by a fragment of the coding region of a reporter gene, and methods for preparing a vector.
Timeline
Filed
05/28/2026
Published
09/17/2026
Granted
Not Available
IPC Codes(5)
A61K 48/00:Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61K 38/00:Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K 31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K 31/00; ergoline-based peptides A61K 31/48; containing macromolecular compounds having statistically distributed amino acid units A61K 31/74; medicinal preparations containing antigens or antibodies A61K 39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K 47/00)
A61P 27/02:Ophthalmic agents